Status:
COMPLETED
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
Lead Sponsor:
Nationwide Children's Hospital
Collaborating Sponsors:
AskBio Inc
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
5-15 years
Phase:
PHASE1
Brief Summary
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
Detailed Description
This phase I randomized double blind dose escalation study investigates the safety and efficacy of the mini-dystrophin gene transferred to the biceps muscle for Duchenne muscular dystrophy patients, a...
Eligibility Criteria
Inclusion
- Known null mutation of the Dystrophin gene
- Male age of 5 years or older
- If taking corticosteroids, must have dose unchanged for the past 3 months
- Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital)
- Progressive, symmetrical proximal muscle weakness of arms and legs
Exclusion
- Unable to cooperate for muscle strength testing
- Joint contractures that prohibit muscle strength testing
- Concomitant illness
- Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension)
- Controlled substance abuse
Key Trial Info
Start Date :
March 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2010
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT00428935
Start Date
March 1 2006
End Date
July 1 2010
Last Update
February 5 2013
Active Locations (1)
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1
Columbus Children's Hospital
Columbus, Ohio, United States, 43205